Event Report: What does it take for Europe to stay at the forefront of rare diseases innovation?

In the past two decades, a combination of scientific breakthrough coupled with strong incentives systems brought about great progress for rare disease patients. The number of medicines approved in Europe to treat rare diseases since 2000 increased from 8 to 169. However, with 95% of rare diseases conditions still without treatment, the need for science and innovation to tackle rare diseases is far from decreasing.

In the lead up to the publication of the OMP and Paediatric Regulations evaluation, EUCOPE brought together a multi-stakeholder dialogue to reflect on this important question: How can Europe remain at the forefront of innovation to the benefit of rare diseases patients?

Introducing the event, Alexander Natz, EUCOPE’s Secretary General, elaborated on EUCOPE’s priorities:

“ The OMP Regulation is crucial to stimulate R&D, bring new treatments to patients and incentivise companies to invest in OMPs. While the regulation has done a great deal to foster the development of OMPs, competitiveness in Europe is decreasing which might lead to postponing innovation, which will be detrimental to patients, especially those living with a rare disease. The EU simply cannot afford this innovation blackhole.”

A summary of the discussion among speakers and key takeaways of the event can be found here.

Building on the event, we partnered with EURACTIV for a series of articles further exploring key issues that we believe need to be addressed to strengthen the EU environment for the development of OMPs. These include the importance of data to close the ‘innovation blackhole’ for rare diseases, the untapped potential of early stakeholder dialogue for the success of rare orphan medicines and finally a reflection encompassing lessons from the past and future outlooks for Europe to remain at the forefront of rare diseases research and innovation.

The entire compilation of articles is available here.

The event, including the panel discussion with the European Commission, industry and patient groups can be watched here.